Leukocyte Adhesion Deficiency-I (LAD-I)

BLA submitted to FDA for RP-L201.  Based on the positive efficacy and safety data from the pivotal study of RP-L201, Rocket submitted the BLA to the FDA, as its first product filing.

Positive, updated top-line data demonstrated 100% overall survival at 12 months post-infusion (and for entire duration of follow-up) via Kaplan Meier estimate for all nine LAD-I patients with 12 to 24 months of available follow-up. Data also showed evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety profile was highly favorable in all patients with no RP-L201-related serious adverse events.